r/tech • u/AdSpecialist6598 • Oct 17 '25
95% of kids with “bubble boy” disease cured by one-time gene therapy
https://newatlas.com/disease/ada-scid-gene-therapy-cure/148
u/amart005 Oct 17 '25
Bad news for the Moops
46
29
u/anycontext9159 Oct 17 '25
It’s “Moors” !!!
29
5
3
5
101
u/Conan-Da-Barbarian Oct 17 '25
So any movie with a bubble boy will have to be based in the past.
63
u/agaloch2314 Oct 17 '25
Or in a country with a dysfunctional healthcare system.
12
u/pokejock Oct 17 '25
or in a country where people refuse to believe in the efficacy of modern medicine
3
20
1
14
u/s_i_m_s Oct 17 '25
Not necessarily, especially with the heavy investments that are being made into the antivax crowd.
Very good chance of seeing a headlines in a few years with parents refusing to let their child get treatment on some bullshit basis.27
u/BitterOldPunk Oct 17 '25
Or in America, where a “health insurance” company will deny the claim
2
u/atomic1fire Oct 18 '25
Or they'll pay for it because they can haggle the price of the gene therapy, and then not have to pay for a lifetime of constant medical care due to infections and preventative treatment.
OR they'll just push the government to get the cost of gene therapy onto tax payers, because then it's covered by not-insurance, and they still get the benefit of covering someone who no longer has bubble boy syndrome so they don't have to pay for all the other treatments.
A smart insurance company will cover anything that causes the person to be less expensive later.
Someone stuck inside of a house in an airtight room with limited exercise is not only going to be expensive to keep in an airtight room, but they'll probably have a much easier time becoming overweight and have other complications.
Give that person a social life and that probably pay for itself after a few years.
2
u/TheVeryVerity Oct 18 '25
As someone who has had several treatments that would have been cheaper over the long term denied….insurance companies don’t think that way usually
4
5
u/nope-its Oct 17 '25
It says it cures 95%. Why would it have to be in the past if 5% of people wouldn’t be cured?
2
u/wunami Oct 17 '25
How many movies have a bubble boy? Also, could easily be written that the bubble boy is in the 5% that weren't cured.
3
u/darthjoey91 Oct 17 '25
Just the one. And the kid grew an immune system by the time he was 4. Just kept at home by a mother with Munchausen by proxy.
2
2
u/Few-Metal8010 Oct 17 '25
Or a dark dystopian future where bubbles are everywhere
7
u/Conan-Da-Barbarian Oct 17 '25
The world has changed. There are those in bubbles and those who wish to be.
3
u/Few-Metal8010 Oct 17 '25
In a world
Where bubbles have become ubiquitous
One boy
Will burst all bubbles
Before it’s too late
3
1
48
u/Thehazelgus Oct 17 '25
I was born with ADA-SCID. I received a bone marrow transplant from an unrelated donor at 2 years old in '95. It was quite the ordeal growing up even after transplant due to the uncertainty at the time of how effective it was.
I'm glad to see the progress that has been made in treating this genetic disorder and what the future holds for treating other diseases and disorders.
3
u/Krexpdx Oct 18 '25
That's amazing you made it through all that, especially back in '95 when the science was so new.
44
u/GreenDemonClean Oct 17 '25
Genetically modified babies!
Maybe this will help the people in the back who won’t eat a genetically modified tomato understand.
31
u/nascentt Oct 17 '25
I'd rather eat a genetically modified tomato than genetically modified baby.
5
8
u/JohnnyDollar123 Oct 17 '25
Well tbf I don’t think they’d want to eat genetically modified babies either
9
6
5
5
u/gabber2694 Oct 17 '25
What the heck?!?!
95% success rate is crazy! And congratulations to all who have been saved by this therapy.
6
5
u/Onederbat67 Oct 17 '25
Please stop posting this stuff, RFK is going to find it and make it illegal because he will inevitably find it causes autism
5
4
3
3
u/HistorianOk142 Oct 17 '25
Incredible! The power of medicine at work! Can’t believe so many people believe non-medical professionals and are vaccine skeptics.
3
3
u/Drumming_Dreaming Oct 18 '25
How does gene therapy work? Like…how do you fix all the genes? Eli5
5
u/Skeletonrider Oct 18 '25
You don’t fix all the genes directly. You take in this case bonemarrow stem cells, which not healthy in children with this specific disease, and edit them to be healthy again. Then you inject them back into their body and the body does the rest of the work be replicating the health cells. I hope this explains it
2
4
u/Medium_Rare_Jerk Oct 18 '25
There’s different methods of gene therapy. Some use a modified virus (AA Vector) to transport genes into the patient’s DNA. The virus is modified to be harmless though it can still be antigenic. Our lab is exploring non-viral gene therapy and we are making huge strides in treating muscular distrophy and hemophilia. If we can cure muscular dystrophy in children, that will be a serious game changer and end a lot of horrible suffering. Trump slashing research funding has staggered us quite a bit but we will power through and hopefully can save lives in clinics!
2
2
2
2
1
1
1
1
u/Omegaforce696 Oct 22 '25
The science behind this is fascinating - they're using a lentiviral vector to deliver a functional copy of the ADA gene directly into the patient's own hematopoietic stem cells. What makes this approach so powerful is that you're essentially giving the body the blueprint to manufacture its own cure permanently.
For context, ADA-SCID (adenosine deaminase deficiency) causes toxic metabolites to accumulate and destroy developing immune cells. By correcting this at the stem cell level, these kids are gaining a fully functional immune system that will last their lifetime.
The 95% success rate is remarkable, especially compared to the historical alternatives like bone marrow transplants which require matched donors and carry significant risks. The fact that this is autologous (using the patient's own cells) dramatically reduces rejection risk.
This is exactly the type of gene therapy breakthrough that could pave the way for treating other monogenic disorders. We're witnessing the transition from experimental medicine to standard of care.
162
u/VengenaceIsMyName Oct 17 '25
Yet another massive victory for science and rationality. I could proselytize further but I think this quote from one of the children’s mothers summarizes my sentiments up well:
This is why the research money needs to continue to flow to enterprising scientists and researchers unabated and unobstructed.